Needless to say, pediatric cancer therapy, especially leukemia, had remarkably progressed and successful changed in last century. However, the chemo-radiation therapy of diffuse intrinsic pontine glioma (DIPG) has still been behind the success story of pediatric cancer therapy. Although, neurosurgical pioneers had tried a lot of new project to cure pediatric DPIG, nothing of new effective protocols have been accepted with satisfactory long term survival. So, we review the pediatric DIPG therapies of neurosurgical pioneers, reconsider temozolomide based protocol, discuss to need brain biopsy, and look for the new direction of pediatric DIPG in the near future.

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  多発性硬化症の診断は，病巣が単発性の場合，しばしば脳腫瘍や脳炎・脳症との鑑別が困難になる．今回われわれは延髄に2 cm大の腫瘤性病変を認め，当初脳幹部グリオーマが疑われたが，最終的に多発性硬化症の診断に至った11歳男児例を経験した．患児は下肢痛の出現後，約2週間の経過で四肢麻痺，意識障害，呼吸不全が進行した．急性の臨床経過がグリオーマの臨床経過と合致せず，診断が困難であったため，手術自体の危険性を説明の上，組織生検を施行した．組織像では明らかな腫瘍細胞を確認しなかったこと，及び症状が急性に進行していることから非腫瘍性疾患の可能性を考え，ステロイドパルス療法を施行したところ速やかに回復し，ほぼ障害を残さずに退院した．その後初発から9か月後に他の部位に再発し，臨床的に多発性硬化症の診断に至った．脳幹部の組織生検は容易ではないが，適切な治療法選択の上で極めて重要な役割を果たした．

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The Japanese Orphan Drug Development Support Program was established in 1993. During the 20 years after its establishment, the Ministry of Health, Labour and Welfare (MHLW) has designated 300 medicines and 23 medical devices as orphan. Of these, 192 medicines and 13 medical devices have been approved. Over the last 10 years from April 2003 to March 2013, 97 medicines were approved including 27 medicines for rare diseases, 17 anticancer therapeutic medicines, 13 medicines for HIV/AIDS and 10 medicines to meet multiple medical needs. The appreciable outcomes of these medicines are as follows. Tocilizumab (genetic recombination) is a humanized anti-human interleukin-6 receptor monoclonal antibody of the IGG1 subclass. It was discovered by Osaka University and Chugai Pharmaceutical Co., Ltd., and was approved for improvement of various symptoms associated with Castleman’s disease in 2005. Pirfenidone was approved for idiopathic pulmonary fibrosis in 2008. Mogamulizumab (genetic recombination), a humanized anti-CCR4 monoclonal antibody, was approved for relapsed or refractory CCR4-positive adult T-cell leukemia/lymphoma in 2012. The above three medicines first commenced in Japan. Pegaptanib sodium was approved for subfoveal choroidal neovascularization secondary to age-related muscular degeneration in 2008. Four alum-adjuvanted influenza (H5 N1) prepandemic vaccines were approved for avian influenza pandemic preparedness in 2007, 2010 and 2013. Regarding medical devices, nine medical devices were approved. For example, an implantable ventricular assist device was approved in 2010. It is used to improve the blood circulation until cardiac transplantation is performed in patients who have severe heart failure for which cardiac transplantation is indicated. Since rare diseases are a global issue, working with international partners and sharing information on orphan drugs have been recognized as issues. This includes organizations such as the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA)． The network involving patients and members of the rare diseases community has been improved. For example, the Cancer Philosophy Clinic was set up as a non-profit organization in 2011. Recently, 31 sites have been opened to care for terminally ill cancer patients. Finally, future proposals for the next decade are also outlined.

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